Behind the recent FDA approval of the first treatment for hyperphagia in PWS is a powerful story of patient data and community collaboration—driven by the Global PWS Registry. Through our PATH for PWS study, families shared their lived experiences with hyperphagia and life with PWS. That data played a critical role in demonstrating to the FDA the urgent need for new treatment options—helping secure approval of VYKAT™ XR for individuals with PWS ages 4 and older.
This is a historic milestone as the Global PWS Registry is the first rare disease registry on the IAMRARE platform to directly support an FDA drug approval!
When Data Drives Progress
Hyperphagia is one of the most difficult and life-limiting aspects of PWS—and until now, no treatment had been approved to address it.
To change that, strong clinical trial data needed to be paired with real-world insights about how hyperphagia impacts daily life. That’s where the Global PWS Registry made a difference.
Through the PATH for PWS study within the registry, families shared data that helped the FDA understand the true burden of hyperphagia and the potential impact of a new treatment. Participating caregivers completed surveys about their loved ones ' hunger-related behaviors. This real-world evidence showed that individuals with PWS who were not taking DCCR (VYKAT XR) had higher hyperphagia scores than those who participated in the Phase 3 DCCR trial — reinforcing the drug’s effectiveness. These patient-reported insights became a key part of the evidence supporting approval of VYKAT XR.
"For years, families and researchers have worked toward a treatment option that truly addresses the complexities of PWS,” said Susan Hedstrom, Executive Director of FPWR. “The IAMRARE platform, created 15 years ago by the National Organization for Rare Disorders (NORD), allowed us to collect the data our community knew was essential—data that helped the FDA fully understand how life-changing a treatment for hyperphagia could be."
The Power of IAMRARE
The IAMRARE patient registry platform, developed by NORD, enables rare disease communities to create and manage patient-driven registries designed to accelerate research and therapeutic development. Developed in collaboration with clinicians, researchers, and the FDA, the platform ensures that the data collected is both meaningful to families and actionable for regulatory and research partners.
As of the end of 2024, IAMRARE hosted 47 patient registries representing approximately 165 rare diseases, including the Global PWS Registry.
This approval marks one of the first times that patient-reported data from an IAMRARE registry played a direct role in supporting an FDA approval—a model that NORD hopes will continue to empower rare disease communities in the future.
Your Participation Matters—Now More Than Ever
The FDA approval of VYKAT XR is a landmark achievement for the PWS community and none of this would have been possible without the families who enrolled in the Global PWS Registry and completed surveys as part of the PATH for PWS study. Your willingness to share your journey helped regulators understand the real-world need for this therapy and contributed to this breakthrough.
The success of this effort demonstrates the value of participating in the Global PWS Registry—not just for advancing scientific understanding, but for shaping the future of treatment for PWS.
If you are already enrolled, thank you! If you haven’t yet joined, or if it’s time to complete new surveys, we encourage you to take part. Your input will help guide the next wave of research and therapeutic development for PWS.
Learn more and sign up at www.pwsregistry.org.
